{"id":1648,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"http:\/\/carola.wwwnl1-sr12.supercp.com\/?p=1648"},"modified":"2023-01-13T11:44:56","modified_gmt":"2023-01-13T11:44:56","slug":"fenotipo-reversible-ratones-humanizados","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/es\/fenotipo-reversible-ratones-humanizados\/","title":{"rendered":"S\u00edntomas reversibles en ratones humanizados"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Shao et al. del grupo del Prof. Zoghbi (Baylor College of Medicine, Texas, EE.UU.) han publicado un art\u00edculo en la revista Science Translational Medicine titulado \"Antisense oligonucleotide therapy in a humanised mouse model of MECP2 duplication syndrome\". <\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Cada especie tiene sus propios genes. Los genes codifican prote\u00ednas espec\u00edficas de cada especie. Las prote\u00ednas humanas y murinas pueden ser ligeramente diferentes. El modelo utilizado en el trabajo presentado, conocido como \"modelo humanizado\", permite evaluar la actividad de los oligonucle\u00f3tidos antisentido (ASO) espec\u00edficamente sobre la prote\u00edna humana producida por un rat\u00f3n. Por tanto, se acerca m\u00e1s al futuro uso de ASO en humanos.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Este art\u00edculo presenta los resultados obtenidos tras la inyecci\u00f3n intracerebroventricular de ASO en el modelo de rat\u00f3n de duplicaci\u00f3n del gen \"MECP2 humanizado\", en el que el rat\u00f3n porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno de rat\u00f3n.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>La administraci\u00f3n redujo eficazmente la expresi\u00f3n de MeCP2 en todo el cerebro de estos ratones, atenu\u00f3 varios d\u00e9ficits de comportamiento y restaur\u00f3 la expresi\u00f3n de ciertos genes regulados por MeCP2 de forma dependiente de la dosis y sin toxicidad alguna.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Terapia con oligonucle\u00f3tidos antisentido en un modelo de rat\u00f3n humanizado del s\u00edndrome de duplicaci\u00f3n MECP2, Shao et al.<em>Sci. Transl. Med<\/em>. <strong>13<\/strong>, 7785 (2021)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RESUMEN DEL ART\u00cdCULO :<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Muchos trastornos que conducen a la discapacidad intelectual se deben a variaciones en el n\u00famero de copias y, hasta la fecha, no se han probado opciones terap\u00e9uticas para esta clase de enfermedades. El s\u00edndrome de duplicaci\u00f3n MECP2 es uno de los reordenamientos gen\u00f3micos m\u00e1s comunes en varones y es el resultado de duplicaciones que abarcan el locus del gen de la prote\u00edna de uni\u00f3n metil-CpG 2 (MECP2). Anteriormente hemos demostrado que la terapia con oligonucle\u00f3tidos antisentido (ASO) puede reducir la cantidad de prote\u00edna MECP2 en un modelo de rat\u00f3n con la duplicaci\u00f3n y revertir los s\u00edntomas caracter\u00edsticos del s\u00edndrome.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sin embargo, este modelo de rat\u00f3n era portador tanto de un alelo transg\u00e9nico humano como de un alelo de rat\u00f3n, estando este \u00faltimo protegido de la acci\u00f3n del ASO-MECP2 espec\u00edfico para humanos.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">La MeCP2 es una prote\u00edna con una concentraci\u00f3n elevada. Por lo tanto, el ASO debe ser titulado para que la cantidad de MeCP2 no sea demasiado baja, ya que esto causar\u00eda el s\u00edndrome de Rett. Por lo tanto, generamos un modelo de duplicaci\u00f3n del gen \"MECP2 humanizado\" en el que el rat\u00f3n porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno del rat\u00f3n. La inyecci\u00f3n intracerebroventricular de ASO-MECP2 redujo eficazmente la expresi\u00f3n de MeCP2 en todo el cerebro de estos ratones. Adem\u00e1s, ASO-MECP2 atenu\u00f3 varios d\u00e9ficits de comportamiento y restaur\u00f3 la expresi\u00f3n de ciertos genes regulados por MeCP2 de forma dependiente de la dosis y sin ninguna toxicidad. La administraci\u00f3n de ASO-MECP2 al sistema nervioso central es, por tanto, bien tolerada y beneficiosa en este modelo de rat\u00f3n y constituye un enfoque transponible que podr\u00eda considerarse para el tratamiento de los s\u00edndromes de duplicaci\u00f3n del gen MECP2.<\/p>\n\n\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button is-style-fill\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/33658357\/\" style=\"border-radius:14px;background-color:#f7a13f\" target=\"_blank\" rel=\"noreferrer noopener\">Ver la publicaci\u00f3n<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Shao et al. du groupe du Pr. Zoghbi (Baylor College of Medicine, Texas, USA) ont publi\u00e9 un article dans la revue Science Translational medicine intitul\u00e9 \u00abTh\u00e9rapie par oligonucl\u00e9otides antisens dans [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11722,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[8],"tags":[],"class_list":["post-1648","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-publications"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Sympt\u00f4mes r\u00e9versibles chez les souris humanis\u00e9es - DupMECP2<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/dupmecp2.eu\/es\/fenotipo-reversible-ratones-humanizados\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Sympt\u00f4mes r\u00e9versibles chez les souris humanis\u00e9es - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"Shao et al. du groupe du Pr. 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